AU2011215557B2 - Targeted genomic modification with partially single-stranded donor molecules - Google Patents
Targeted genomic modification with partially single-stranded donor molecules Download PDFInfo
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- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
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- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
- C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
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PCT/US2011/000245 WO2011100058A1 (en) | 2010-02-09 | 2011-02-09 | Targeted genomic modification with partially single-stranded donor molecules |
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US20120196370A1 (en) | 2010-12-03 | 2012-08-02 | Fyodor Urnov | Methods and compositions for targeted genomic deletion |
US20110239315A1 (en) | 2009-01-12 | 2011-09-29 | Ulla Bonas | Modular dna-binding domains and methods of use |
EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
US8399643B2 (en) | 2009-02-26 | 2013-03-19 | Transposagen Biopharmaceuticals, Inc. | Nucleic acids encoding hyperactive PiggyBac transposases |
US8586363B2 (en) | 2009-12-10 | 2013-11-19 | Regents Of The University Of Minnesota | TAL effector-mediated DNA modification |
EP2533629B1 (en) * | 2010-02-11 | 2018-11-28 | Recombinetics, Inc. | Methods and materials for producing transgenic artiodactyls |
EP3156062A1 (en) | 2010-05-17 | 2017-04-19 | Sangamo BioSciences, Inc. | Novel dna-binding proteins and uses thereof |
EP2392208B1 (en) * | 2010-06-07 | 2016-05-04 | Helmholtz Zentrum München Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) | Fusion proteins comprising a DNA-binding domain of a Tal effector protein and a non-specific cleavage domain of a restriction nuclease and their use |
US10920242B2 (en) | 2011-02-25 | 2021-02-16 | Recombinetics, Inc. | Non-meiotic allele introgression |
US9528124B2 (en) | 2013-08-27 | 2016-12-27 | Recombinetics, Inc. | Efficient non-meiotic allele introgression |
WO2013016446A2 (en) | 2011-07-25 | 2013-01-31 | Sangamo Biosciences, Inc. | Methods and compositions for alteration of a cystic fibrosis transmembrane conductance regulator (cftr) gene |
CN103917644A (zh) | 2011-09-21 | 2014-07-09 | 桑格摩生物科学股份有限公司 | 调控转基因表达的方法和组合物 |
JP6188703B2 (ja) | 2011-10-27 | 2017-08-30 | サンガモ セラピューティクス, インコーポレイテッド | Hprt遺伝子座を修飾するための方法および組成物 |
GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
DK2839013T3 (da) * | 2012-04-18 | 2020-09-14 | Univ Leland Stanford Junior | Ikke-disruptiv-gen-targetering |
ES2683071T3 (es) * | 2012-04-25 | 2018-09-24 | Regeneron Pharmaceuticals, Inc. | Direccionamiento mediado por nucleasas con grandes vectores de direccionamiento |
AU2013256240B2 (en) | 2012-05-02 | 2018-09-20 | Corteva Agriscience Llc | Targeted modification of malate dehydrogenase |
CN104471067B (zh) * | 2012-05-07 | 2020-08-14 | 桑格摩生物治疗股份有限公司 | 用于核酸酶介导的转基因靶向整合的方法和组合物 |
DK2800811T3 (en) | 2012-05-25 | 2017-07-17 | Univ Vienna | METHODS AND COMPOSITIONS FOR RNA DIRECTIVE TARGET DNA MODIFICATION AND FOR RNA DIRECTIVE MODULATION OF TRANSCRIPTION |
WO2013182910A2 (en) * | 2012-06-05 | 2013-12-12 | Cellectis | New transcription activator-like effector (tale) fusion protein |
BR112014031891A2 (pt) | 2012-06-19 | 2017-08-01 | Univ Minnesota | direcionamento genético nas plantas utilizando vírus de dna |
WO2013191769A1 (en) * | 2012-06-22 | 2013-12-27 | Mayo Foundation For Medical Education And Research | Genome editing |
EP3196301B1 (en) * | 2012-07-11 | 2018-10-17 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of monogenic diseases |
US10648001B2 (en) | 2012-07-11 | 2020-05-12 | Sangamo Therapeutics, Inc. | Method of treating mucopolysaccharidosis type I or II |
JP6329537B2 (ja) * | 2012-07-11 | 2018-05-23 | サンガモ セラピューティクス, インコーポレイテッド | 生物学的薬剤の送達のための方法および組成物 |
JP2015527889A (ja) * | 2012-07-25 | 2015-09-24 | ザ ブロード インスティテュート, インコーポレイテッド | 誘導可能なdna結合タンパク質およびゲノム撹乱ツール、ならびにそれらの適用 |
US10058078B2 (en) | 2012-07-31 | 2018-08-28 | Recombinetics, Inc. | Production of FMDV-resistant livestock by allele substitution |
WO2014033644A2 (en) | 2012-08-28 | 2014-03-06 | Novartis Ag | Methods of nuclease-based genetic engineering |
DK2890780T3 (da) | 2012-08-29 | 2020-09-21 | Sangamo Therapeutics Inc | Fremgangsmåder og sammensætninger til behandling af en genetisk tilstand |
UA118090C2 (uk) | 2012-09-07 | 2018-11-26 | ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі | Спосіб інтегрування послідовності нуклеїнової кислоти, що представляє інтерес, у ген fad2 у клітині сої та специфічний для локусу fad2 білок, що зв'язується, здатний індукувати спрямований розрив |
JP6775953B2 (ja) | 2012-09-07 | 2020-10-28 | ダウ アグロサイエンシィズ エルエルシー | Fad3性能座および標的化切断を誘導可能である対応する標的部位特異的結合タンパク質 |
GB201216564D0 (en) | 2012-09-17 | 2012-10-31 | Univ Edinburgh | Genetically edited animal |
EP3763810A3 (en) | 2012-10-10 | 2021-07-14 | Sangamo Therapeutics, Inc. | T cell modifying compounds and uses thereof |
BR112015009967B1 (pt) | 2012-11-01 | 2022-07-12 | Medicago Inc. | Método para a produção de um polipeptídeo terapêutico |
US9255250B2 (en) | 2012-12-05 | 2016-02-09 | Sangamo Bioscience, Inc. | Isolated mouse or human cell having an exogenous transgene in an endogenous albumin gene |
KR102243092B1 (ko) | 2012-12-06 | 2021-04-22 | 시그마-알드리치 컴퍼니., 엘엘씨 | Crispr-기초된 유전체 변형과 조절 |
WO2014093709A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
SG11201504523UA (en) | 2012-12-12 | 2015-07-30 | Broad Inst Inc | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
ES2673864T3 (es) | 2012-12-21 | 2018-06-26 | Cellectis | Patatas con endulzamiento inducido en frío reducido |
WO2014102688A1 (en) * | 2012-12-27 | 2014-07-03 | Cellectis | New design matrix for improvement of homology-directed gene targeting |
US10227610B2 (en) | 2013-02-25 | 2019-03-12 | Sangamo Therapeutics, Inc. | Methods and compositions for enhancing nuclease-mediated gene disruption |
EP2971167B1 (en) | 2013-03-14 | 2019-07-31 | Caribou Biosciences, Inc. | Compositions and methods of nucleic acid-targeting nucleic acids |
ES2786253T3 (es) | 2013-03-14 | 2020-10-09 | Immusoft Corp | Métodos para la diferenciación y transducción in vitro de células b de memoria con vectores virales pseudotipados vsv-g |
US10113162B2 (en) | 2013-03-15 | 2018-10-30 | Cellectis | Modifying soybean oil composition through targeted knockout of the FAD2-1A/1B genes |
US9957515B2 (en) | 2013-03-15 | 2018-05-01 | Cibus Us Llc | Methods and compositions for targeted gene modification |
WO2014153470A2 (en) | 2013-03-21 | 2014-09-25 | Sangamo Biosciences, Inc. | Targeted disruption of t cell receptor genes using engineered zinc finger protein nucleases |
KR102192599B1 (ko) | 2013-04-05 | 2020-12-18 | 다우 아그로사이언시즈 엘엘씨 | 식물의 게놈 내의 외인성 서열의 통합을 위한 방법 및 조성물 |
RS62263B1 (sr) | 2013-04-16 | 2021-09-30 | Regeneron Pharma | Ciljana modifikacija genoma pacova |
AU2014262867B2 (en) | 2013-05-10 | 2019-12-05 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
CN116083487A (zh) | 2013-05-15 | 2023-05-09 | 桑格摩生物治疗股份有限公司 | 用于治疗遗传病状的方法和组合物 |
JP6387400B2 (ja) * | 2013-05-30 | 2018-09-05 | ナノビオティックスNanobiotix | 医薬組成物、その製造および使用 |
CA2915842C (en) | 2013-06-17 | 2022-11-29 | The Broad Institute, Inc. | Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy |
EP4245853A3 (en) * | 2013-06-17 | 2023-10-18 | The Broad Institute, Inc. | Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation |
CN106062197A (zh) | 2013-06-17 | 2016-10-26 | 布罗德研究所有限公司 | 用于序列操纵的串联指导系统、方法和组合物的递送、工程化和优化 |
KR20160056869A (ko) | 2013-06-17 | 2016-05-20 | 더 브로드 인스티튜트, 인코퍼레이티드 | 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용 |
WO2014204727A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
ES2915377T3 (es) | 2013-08-02 | 2022-06-22 | Enevolv Inc | Procedimientos y células huésped para ingeniería genómica, de vías y biomolécular |
EP3988654A1 (en) | 2013-08-28 | 2022-04-27 | Sangamo Therapeutics, Inc. | Compositions for linking dna-binding domains and cleavage domains |
EP3057432B1 (en) | 2013-10-17 | 2018-11-21 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells |
ES2881473T3 (es) | 2013-10-17 | 2021-11-29 | Sangamo Therapeutics Inc | Métodos de suministro y composiciones para la modificación por ingeniería genética del genoma mediada por nucleasas |
AP2016009207A0 (en) | 2013-10-25 | 2016-05-31 | Livestock Improvement Corp Ltd | Genetic markers and uses therefor |
EP3066109A4 (en) | 2013-11-04 | 2017-11-29 | Dow AgroSciences LLC | Optimal soybean loci |
NZ746567A (en) | 2013-11-04 | 2019-09-27 | Dow Agrosciences Llc | Optimal soybean loci |
KR102269769B1 (ko) * | 2013-11-04 | 2021-06-28 | 코르테바 애그리사이언스 엘엘씨 | 최적 메이즈 유전자좌 |
CA2928855A1 (en) | 2013-11-04 | 2015-05-07 | Dow Agrosciences Llc | Optimal maize loci |
PL3492593T3 (pl) | 2013-11-13 | 2022-04-19 | Children's Medical Center Corporation | Regulacja ekspresji genów, w której pośredniczą nukleazy |
CA2930590C (en) * | 2013-11-15 | 2021-02-16 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Engineering neural stem cells using homologous recombination |
EP3757116A1 (en) | 2013-12-09 | 2020-12-30 | Sangamo Therapeutics, Inc. | Methods and compositions for genome engineering |
RU2725520C2 (ru) | 2013-12-11 | 2020-07-02 | Регенерон Фармасьютикалс, Инк. | Способы и композиции для направленной модификации генома |
WO2015089462A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing |
WO2015089364A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crystal structure of a crispr-cas system, and uses thereof |
CA2932472A1 (en) | 2013-12-12 | 2015-06-18 | Massachusetts Institute Of Technology | Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders |
JP6793547B2 (ja) | 2013-12-12 | 2020-12-02 | ザ・ブロード・インスティテュート・インコーポレイテッド | 最適化機能CRISPR−Cas系による配列操作のための系、方法および組成物 |
US10774338B2 (en) | 2014-01-16 | 2020-09-15 | The Regents Of The University Of California | Generation of heritable chimeric plant traits |
US10233456B2 (en) | 2014-01-30 | 2019-03-19 | The Board Of Trustees Of The University Of Arkansas | Method, vectors, cells, seeds and kits for stacking genes into a single genomic site |
WO2015117081A2 (en) | 2014-02-03 | 2015-08-06 | Sangamo Biosciences, Inc. | Methods and compositions for treatment of a beta thalessemia |
AU2015218576B2 (en) | 2014-02-24 | 2020-02-27 | Sangamo Therapeutics, Inc. | Methods and compositions for nuclease-mediated targeted integration |
IL247679B1 (en) * | 2014-03-14 | 2024-07-01 | Cibus Europe Bv | Methods and compositions for increasing the efficiency of targeted gene modification through the use of oligonucleotide-mediated gene repair |
CA2942762C (en) | 2014-03-18 | 2023-10-17 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of zinc finger protein expression |
AU2015231231B2 (en) | 2014-03-21 | 2021-09-02 | The Board Of Trustees Of The Leland Stanford Junior University | Genome editing without nucleases |
US9522936B2 (en) | 2014-04-24 | 2016-12-20 | Sangamo Biosciences, Inc. | Engineered transcription activator like effector (TALE) proteins |
CN111647627A (zh) * | 2014-04-28 | 2020-09-11 | 重组股份有限公司 | 多重基因编辑 |
CN106460050A (zh) * | 2014-04-28 | 2017-02-22 | 西格马-奥尔德里奇有限责任公司 | 使用靶向核酸内切酶进行哺乳动物基因组的表观遗传修饰 |
GB201407852D0 (en) | 2014-05-02 | 2014-06-18 | Iontas Ltd | Preparation of libraries od protein variants expressed in eukaryotic cells and use for selecting binding molecules |
EP3142707A4 (en) | 2014-05-13 | 2018-02-21 | Sangamo Therapeutics, Inc. | Methods and compositions for prevention or treatment of a disease |
WO2015188065A1 (en) | 2014-06-05 | 2015-12-10 | Sangamo Biosciences, Inc. | Methods and compositions for nuclease design |
EP3708671A1 (en) | 2014-06-06 | 2020-09-16 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for modifying a targeted locus |
WO2015193858A1 (en) | 2014-06-20 | 2015-12-23 | Cellectis | Potatoes with reduced granule-bound starch synthase |
CA2953362A1 (en) * | 2014-06-23 | 2015-12-30 | The General Hospital Corporation | Genomewide unbiased identification of dsbs evaluated by sequencing (guide-seq) |
US9902971B2 (en) | 2014-06-26 | 2018-02-27 | Regeneron Pharmaceuticals, Inc. | Methods for producing a mouse XY embryonic (ES) cell line capable of producing a fertile XY female mouse in an F0 generation |
ES2965286T3 (es) | 2014-07-14 | 2024-04-12 | Univ Washington State | Silenciamiento de NANOS que abate las células germinales |
WO2016014794A1 (en) | 2014-07-25 | 2016-01-28 | Sangamo Biosciences, Inc. | Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells |
US9757420B2 (en) | 2014-07-25 | 2017-09-12 | Sangamo Therapeutics, Inc. | Gene editing for HIV gene therapy |
WO2016019144A2 (en) | 2014-07-30 | 2016-02-04 | Sangamo Biosciences, Inc. | Gene correction of scid-related genes in hematopoietic stem and progenitor cells |
MX2017002931A (es) | 2014-09-07 | 2017-05-30 | Selecta Biosciences Inc | Metodos y composiciones para atenuar respuestas inmunes anti-vector de transferencia viral. |
RS62334B1 (sr) | 2014-09-16 | 2021-10-29 | Sangamo Therapeutics Inc | Postupci i sastavi za inženjering genoma posredovan nukleazom i korekciju kod matičnih ćelija hematopoeze |
CA2963820A1 (en) | 2014-11-07 | 2016-05-12 | Editas Medicine, Inc. | Methods for improving crispr/cas-mediated genome-editing |
CN107532142A (zh) * | 2014-11-11 | 2018-01-02 | 应用干细胞有限公司 | 使用同源重组改造间充质干细胞 |
SI3221457T1 (sl) | 2014-11-21 | 2019-08-30 | Regeneron Pharmaceuticals, Inc. | Postopki in sestavki za ciljno genetsko modifikacijo z uporabo vodilnih RNK v parih |
WO2016094867A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Protected guide rnas (pgrnas) |
US10889834B2 (en) | 2014-12-15 | 2021-01-12 | Sangamo Therapeutics, Inc. | Methods and compositions for enhancing targeted transgene integration |
WO2016100819A1 (en) | 2014-12-19 | 2016-06-23 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for targeted genetic modification through single-step multiple targeting |
WO2016118726A2 (en) | 2015-01-21 | 2016-07-28 | Sangamo Biosciences, Inc. | Methods and compositions for identification of highly specific nucleases |
US10179918B2 (en) | 2015-05-07 | 2019-01-15 | Sangamo Therapeutics, Inc. | Methods and compositions for increasing transgene activity |
BR112017024115A2 (pt) | 2015-05-12 | 2018-08-07 | Sangamo Therapeutics Inc | regulação de expressão genética mediada por nuclease |
WO2016205759A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation |
CN109536474A (zh) | 2015-06-18 | 2019-03-29 | 布罗德研究所有限公司 | 降低脱靶效应的crispr酶突变 |
US9957501B2 (en) | 2015-06-18 | 2018-05-01 | Sangamo Therapeutics, Inc. | Nuclease-mediated regulation of gene expression |
EP3322297A4 (en) | 2015-07-13 | 2019-04-17 | Sangamo Therapeutics, Inc. | RELEASE METHOD AND COMPOSITIONS FOR NUCLEASE-DERIVED GENOMINE ENGINEERING |
LT3331355T (lt) | 2015-08-06 | 2024-07-25 | The Curators Of The University Of Missouri | Kiaulių reprodukcinio ir respiracinio sindromo virusui (prrsv) atsparios kiaulės ir ląstelės, turinčios modifikuotus cd163 genus |
WO2017044843A1 (en) | 2015-09-11 | 2017-03-16 | The General Hospital Corporation | Full interrogation of nuclease dsbs and sequencing (find-seq) |
US10837024B2 (en) | 2015-09-17 | 2020-11-17 | Cellectis | Modifying messenger RNA stability in plant transformations |
WO2017053753A1 (en) | 2015-09-23 | 2017-03-30 | Sangamo Biosciences, Inc. | Htt repressors and uses thereof |
US11667911B2 (en) | 2015-09-24 | 2023-06-06 | Editas Medicine, Inc. | Use of exonucleases to improve CRISPR/CAS-mediated genome editing |
CA3000762A1 (en) | 2015-09-30 | 2017-04-06 | The General Hospital Corporation | Comprehensive in vitro reporting of cleavage events by sequencing (circle-seq) |
PE20181206A1 (es) | 2015-10-28 | 2018-07-23 | Sangamo Therapeutics Inc | Construcciones especificas del higado, casetes de expresion del factor viii y metodos de uso de estos |
EP3380622A4 (en) | 2015-11-23 | 2019-08-07 | Sangamo Therapeutics, Inc. | METHODS AND COMPOSITIONS FOR MODIFYING IMMUNITY |
EP3389677B1 (en) | 2015-12-18 | 2024-06-26 | Sangamo Therapeutics, Inc. | Targeted disruption of the t cell receptor |
JP2018537112A (ja) | 2015-12-18 | 2018-12-20 | サンガモ セラピューティクス, インコーポレイテッド | Mhc細胞受容体の標的化破壊 |
WO2017123609A1 (en) * | 2016-01-12 | 2017-07-20 | The Regents Of The University Of California | Compositions and methods for enhanced genome editing |
JP6930052B2 (ja) | 2016-01-15 | 2021-09-01 | サンガモ セラピューティクス, インコーポレイテッド | 神経性疾患の処置のための方法および組成物 |
UY37108A (es) | 2016-02-02 | 2017-08-31 | Cellectis | Modificación de la composición de aceites de soja mediante el knockout dirigido de los genes fad3a/b/c |
EP3411056A4 (en) | 2016-02-02 | 2019-10-02 | Sangamo Therapeutics, Inc. | COMPOUNDS FOR NETWORKING DNA BINDING DOMAINS AND SPLITTING DOMAINS |
US11597924B2 (en) | 2016-03-25 | 2023-03-07 | Editas Medicine, Inc. | Genome editing systems comprising repair-modulating enzyme molecules and methods of their use |
EP4047092A1 (en) | 2016-04-13 | 2022-08-24 | Editas Medicine, Inc. | Cas9 fusion molecules, gene editing systems, and methods of use thereof |
EP3464587B1 (en) * | 2016-05-27 | 2020-12-16 | Life Technologies Corporation | Compositions and methods for enhancing homologous recombination |
EP3907286A1 (en) | 2016-06-02 | 2021-11-10 | Sigma-Aldrich Co., LLC | Using programmable dna binding proteins to enhance targeted genome modification |
WO2018035159A1 (en) | 2016-08-15 | 2018-02-22 | Enevolv, Inc. | Molecule sensor systems |
PE20190568A1 (es) | 2016-08-24 | 2019-04-22 | Sangamo Therapeutics Inc | Regulacion de la expresion genica usando nucleasas modificadas |
KR20220145913A (ko) | 2016-08-24 | 2022-10-31 | 상가모 테라퓨틱스, 인코포레이티드 | 가공된 표적 특이적 뉴클레아제 |
US10960085B2 (en) | 2016-09-07 | 2021-03-30 | Sangamo Therapeutics, Inc. | Modulation of liver genes |
EP3519562A4 (en) | 2016-09-29 | 2020-03-25 | Nantkwest, Inc. | NK-92 HLA CLASS I DEFICIENT CELLS WITH REDUCED IMMUNOGENICITY |
MX2019003768A (es) | 2016-10-03 | 2019-06-24 | Juno Therapeutics Inc | Moleculas de enlace especificas de hpv. |
US11219695B2 (en) | 2016-10-20 | 2022-01-11 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of Fabry disease |
US11020492B2 (en) | 2016-10-31 | 2021-06-01 | Sangamo Therapeutics, Inc. | Gene correction of SCID-related genes in hematopoietic stem and progenitor cells |
WO2018092072A1 (en) | 2016-11-16 | 2018-05-24 | Cellectis | Methods for altering amino acid content in plants through frameshift mutations |
WO2018106782A1 (en) | 2016-12-08 | 2018-06-14 | Case Western Reserve University | Methods and compositions for enhancing functional myelin production |
WO2018129346A1 (en) | 2017-01-06 | 2018-07-12 | Nantkwest, Inc. | Genetically modified nk-92 cells with decreased cd96/tigit expression |
US10457717B2 (en) | 2017-02-17 | 2019-10-29 | Denali Therapeutics Inc. | Engineered polypeptides |
DK3583120T5 (da) | 2017-02-17 | 2024-09-02 | Denali Therapeutics Inc | Modificerede transferrinreceptorbindende polypeptider |
SG11201907419PA (en) | 2017-02-17 | 2019-09-27 | Denali Therapeutics Inc | Transferrin receptor transgenic models |
US10143187B2 (en) | 2017-02-17 | 2018-12-04 | Denali Therapeutics Inc. | Transferrin receptor transgenic models |
ES2977620T3 (es) | 2017-04-25 | 2024-08-27 | Cellectis | Alfalfa con composición reducida de lignina |
IL301115A (en) | 2017-04-28 | 2023-05-01 | Acuitas Therapeutics Inc | New lipid carbonyl and lipid nanoparticle formulations for delivery of nucleic acids |
WO2018204469A2 (en) | 2017-05-03 | 2018-11-08 | Sangamo Therapeutics, Inc. | Methods and compositions for modification of a cystic fibrosis transmembrane conductance regulator (cftr) gene |
US11512287B2 (en) | 2017-06-16 | 2022-11-29 | Sangamo Therapeutics, Inc. | Targeted disruption of T cell and/or HLA receptors |
US11866726B2 (en) | 2017-07-14 | 2024-01-09 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
BR112020003596A2 (pt) | 2017-08-23 | 2020-09-01 | The General Hospital Corporation | nucleases de crispr-cas9 engenheiradas com especificidade de pam alterada |
JP2020537515A (ja) | 2017-10-03 | 2020-12-24 | ジュノー セラピューティクス インコーポレイテッド | Hpv特異的結合分子 |
EP3694993A4 (en) | 2017-10-11 | 2021-10-13 | The General Hospital Corporation | METHOD OF DETECTING A SITE-SPECIFIC AND UNDESIRED GENOMIC DESAMINATION INDUCED BY BASE EDITING TECHNOLOGIES |
WO2019075360A1 (en) | 2017-10-13 | 2019-04-18 | Selecta Biosciences, Inc. | METHODS AND COMPOSITIONS FOR MITIGATING ANTI-VECTOR VIRAL TRANSFER IGM RESPONSES |
US11851679B2 (en) | 2017-11-01 | 2023-12-26 | Juno Therapeutics, Inc. | Method of assessing activity of recombinant antigen receptors |
KR20200079539A (ko) | 2017-11-09 | 2020-07-03 | 상가모 테라퓨틱스, 인코포레이티드 | 시토카인 유도성 sh2-함유 단백질 (cish) 유전자의 유전자 변형 |
EP3728598A1 (en) | 2017-12-21 | 2020-10-28 | Max-Delbrück-Centrum für Molekulare Medizin in der Helmholtz-Gemeinschaft | Nucleic acid sequence replacement by nhej |
EP3749350A4 (en) | 2018-02-08 | 2021-12-01 | Sangamo Therapeutics, Inc. | SPECIFICALLY MODIFIED TARGET-SPECIFIC NUCLEASES |
AU2019225251A1 (en) | 2018-02-26 | 2020-10-15 | AnTolRx, Inc. | Tolerogenic liposomes and methods of use thereof |
CA3093716A1 (en) | 2018-03-16 | 2019-09-19 | Immusoft Corporation | B cells genetically engineered to secrete follistatin and methods of using the same to treat follistatin-related diseases, conditions, disorders and to enhance muscle growth and strength |
BR112020020245A2 (pt) | 2018-04-05 | 2021-04-06 | Editas Medicine, Inc. | Métodos de produzir células expressando um receptor recombinante e composições relacionadas |
CA3095084A1 (en) | 2018-04-05 | 2019-10-10 | Juno Therapeutics, Inc. | T cells expressing a recombinant receptor, related polynucleotides and methods |
KR20210019993A (ko) | 2018-04-05 | 2021-02-23 | 주노 쎄러퓨티크스 인코퍼레이티드 | Τ 세포 수용체 및 이를 발현하는 조작된 세포 |
WO2019204378A1 (en) | 2018-04-17 | 2019-10-24 | The General Hospital Corporation | Sensitive in vitro assays for substrate preferences and sites of nucleic acid binding, modifying, and cleaving agents |
US11421007B2 (en) | 2018-04-18 | 2022-08-23 | Sangamo Therapeutics, Inc. | Zinc finger protein compositions for modulation of huntingtin (Htt) |
US11690921B2 (en) | 2018-05-18 | 2023-07-04 | Sangamo Therapeutics, Inc. | Delivery of target specific nucleases |
WO2020014235A1 (en) | 2018-07-09 | 2020-01-16 | The Regents Of The University Of California | Gene targets for t-cell-based immunotherapy |
AU2019326408A1 (en) | 2018-08-23 | 2021-03-11 | Sangamo Therapeutics, Inc. | Engineered target specific base editors |
EP3853244A4 (en) | 2018-09-18 | 2022-09-14 | Sangamo Therapeutics, Inc. | PROGRAMMED CELL DEATH 1 (PD1) SPECIFIC NUCLEASES |
WO2020069029A1 (en) | 2018-09-26 | 2020-04-02 | Emendobio Inc. | Novel crispr nucleases |
CN109161587A (zh) * | 2018-09-26 | 2019-01-08 | 上海交通大学医学院附属上海儿童医学中心 | 一种检测染色体重复片段断裂位点和定位信息的方法 |
MA53919A (fr) | 2018-10-18 | 2021-08-25 | Intellia Therapeutics Inc | Constructions d'acides nucléiques et procédés d'utilisation |
TW202027798A (zh) | 2018-10-18 | 2020-08-01 | 美商英特利亞醫療公司 | 用於從白蛋白基因座表現轉殖基因的組成物及方法 |
MX2021004276A (es) | 2018-10-18 | 2021-09-08 | Intellia Therapeutics Inc | Composiciones y metodos para tratar deficiencia de alfa-1 antitripsina. |
BR112021007301A2 (pt) | 2018-10-18 | 2021-07-27 | Intellia Therapeutics, Inc. | composições e métodos para expressar fator ix |
EP3673920A1 (en) | 2018-12-28 | 2020-07-01 | Universität Wien | Polyphenol-peptide conjugates for nuclear-targeted delivery |
JP7523449B2 (ja) | 2019-01-11 | 2024-07-26 | アクイタス セラピューティクス インコーポレイテッド | 活性剤の脂質ナノ粒子送達のための脂質 |
US20220098621A1 (en) | 2019-02-05 | 2022-03-31 | Emendobio Inc. | Crispr compositions and methods for promoting gene editing of ribosomal protein s19 (rps19) gene |
US11857641B2 (en) | 2019-02-06 | 2024-01-02 | Sangamo Therapeutics, Inc. | Method for the treatment of mucopolysaccharidosis type I |
WO2020163307A1 (en) | 2019-02-06 | 2020-08-13 | Emendobio Inc. | New engineered high fidelity cas9 |
BR112021021200A2 (pt) | 2019-05-01 | 2021-12-21 | Juno Therapeutics Inc | Células expressando um receptor quimérico de um locus cd247 modificado, polinucleotídeos relacionados e métodos |
SG11202111360YA (en) | 2019-05-01 | 2021-11-29 | Juno Therapeutics Inc | Cells expressing a recombinant receptor from a modified tgfbr2 locus, related polynucleotides and methods |
CA3141863A1 (en) | 2019-05-28 | 2020-12-03 | Selecta Biosciences, Inc. | Methods and compositions for attenuated anti-viral transfer vector immune response |
US20210079387A1 (en) * | 2019-08-27 | 2021-03-18 | The Jackson Laboratory | Cleavage-resistant donor nucleic acids and methods of use |
US20240100184A1 (en) * | 2019-10-21 | 2024-03-28 | Rowan University | Methods of precise genome editing by in situ cut and paste (icap) |
EP4150057A2 (en) | 2020-05-13 | 2023-03-22 | Juno Therapeutics, Inc. | Process for producing donor-batched cells expressing a recombinant receptor |
CN116234558A (zh) | 2020-06-26 | 2023-06-06 | 朱诺治疗学有限公司 | 条件性地表达重组受体的工程化t细胞、相关多核苷酸和方法 |
AU2021308681A1 (en) | 2020-07-16 | 2023-03-09 | Acuitas Therapeutics, Inc. | Cationic lipids for use in lipid nanoparticles |
WO2022098787A1 (en) | 2020-11-04 | 2022-05-12 | Juno Therapeutics, Inc. | Cells expressing a chimeric receptor from a modified invariant cd3 immunoglobulin superfamily chain locus and related polynucleotides and methods |
US20240000051A1 (en) | 2020-11-16 | 2024-01-04 | Pig Improvement Company Uk Limited | Influenza a-resistant animals having edited anp32 genes |
CN117321200A (zh) | 2021-03-22 | 2023-12-29 | 朱诺治疗学股份有限公司 | 评估病毒载体颗粒效力的方法 |
TW202305118A (zh) | 2021-03-23 | 2023-02-01 | 美商艾歐凡斯生物治療公司 | 腫瘤浸潤淋巴球之cish基因編輯及其在免疫療法中之用途 |
EP4362957A1 (en) | 2021-07-01 | 2024-05-08 | Indapta Therapeutics, Inc. | Engineered natural killer (nk) cells and related methods |
US20230141563A1 (en) | 2021-10-12 | 2023-05-11 | Selecta Biosciences, Inc. | Methods and compositions for attenuating anti-viral transfer vector igm responses |
US20230149563A1 (en) | 2021-10-27 | 2023-05-18 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for expressing factor ix for hemophilia b therapy |
WO2023081900A1 (en) | 2021-11-08 | 2023-05-11 | Juno Therapeutics, Inc. | Engineered t cells expressing a recombinant t cell receptor (tcr) and related systems and methods |
WO2023105244A1 (en) | 2021-12-10 | 2023-06-15 | Pig Improvement Company Uk Limited | Editing tmprss2/4 for disease resistance in livestock |
KR20240133705A (ko) | 2021-12-17 | 2024-09-04 | 데날리 테라퓨틱스 인크. | 항-pilra 항체, 이의 용도, 및 관련 방법 및 시약 |
WO2023150620A1 (en) | 2022-02-02 | 2023-08-10 | Regeneron Pharmaceuticals, Inc. | Crispr-mediated transgene insertion in neonatal cells |
US20230357437A1 (en) | 2022-03-09 | 2023-11-09 | Selecta Biosciences, Inc. | Immunosuppressants in combination with anti-igm agents and related dosing |
WO2023212677A2 (en) | 2022-04-29 | 2023-11-02 | Regeneron Pharmaceuticals, Inc. | Identification of tissue-specific extragenic safe harbors for gene therapy approaches |
WO2024007020A1 (en) | 2022-06-30 | 2024-01-04 | Indapta Therapeutics, Inc. | Combination of engineered natural killer (nk) cells and antibody therapy and related methods |
WO2024013514A2 (en) | 2022-07-15 | 2024-01-18 | Pig Improvement Company Uk Limited | Gene edited livestock animals having coronavirus resistance |
WO2024100604A1 (en) | 2022-11-09 | 2024-05-16 | Juno Therapeutics Gmbh | Methods for manufacturing engineered immune cells |
WO2024161021A1 (en) | 2023-02-03 | 2024-08-08 | Juno Therapeutics Gmbh | Methods for non-viral manufacturing of engineered immune cells |
Citations (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2010006237A2 (en) * | 2008-07-11 | 2010-01-14 | Alnylam Pharmaceuticals, Inc. | Phosphorothioate oligonucleotides non-nucleosidic phosphorothiotes as delivery agents for irna agents |
WO2010143917A2 (en) * | 2009-06-11 | 2010-12-16 | Toolgen Incorporation | Targeted genomic rearrangements using site-specific nucleases |
Family Cites Families (87)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US4217344A (en) | 1976-06-23 | 1980-08-12 | L'oreal | Compositions containing aqueous dispersions of lipid spheres |
US4235871A (en) | 1978-02-24 | 1980-11-25 | Papahadjopoulos Demetrios P | Method of encapsulating biologically active materials in lipid vesicles |
US4186183A (en) | 1978-03-29 | 1980-01-29 | The United States Of America As Represented By The Secretary Of The Army | Liposome carriers in chemotherapy of leishmaniasis |
US4261975A (en) | 1979-09-19 | 1981-04-14 | Merck & Co., Inc. | Viral liposome particle |
US4485054A (en) | 1982-10-04 | 1984-11-27 | Lipoderm Pharmaceuticals Limited | Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV) |
US4501728A (en) | 1983-01-06 | 1985-02-26 | Technology Unlimited, Inc. | Masking of liposomes from RES recognition |
US5049386A (en) | 1985-01-07 | 1991-09-17 | Syntex (U.S.A.) Inc. | N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
US4946787A (en) | 1985-01-07 | 1990-08-07 | Syntex (U.S.A.) Inc. | N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
US4897355A (en) | 1985-01-07 | 1990-01-30 | Syntex (U.S.A.) Inc. | N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor |
US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
US4774085A (en) | 1985-07-09 | 1988-09-27 | 501 Board of Regents, Univ. of Texas | Pharmaceutical administration systems containing a mixture of immunomodulators |
US5422251A (en) | 1986-11-26 | 1995-06-06 | Princeton University | Triple-stranded nucleic acids |
US4837028A (en) | 1986-12-24 | 1989-06-06 | Liposome Technology, Inc. | Liposomes with enhanced circulation time |
US5176996A (en) | 1988-12-20 | 1993-01-05 | Baylor College Of Medicine | Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use |
FR2646438B1 (fr) | 1989-03-20 | 2007-11-02 | Pasteur Institut | Procede de remplacement specifique d'une copie d'un gene present dans le genome receveur par l'integration d'un gene different de celui ou se fait l'integration |
US5264618A (en) | 1990-04-19 | 1993-11-23 | Vical, Inc. | Cationic lipids for intracellular delivery of biologically active molecules |
WO1991017424A1 (en) | 1990-05-03 | 1991-11-14 | Vical, Inc. | Intracellular delivery of biologically active substances by means of self-assembling lipid complexes |
US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
US5420032A (en) | 1991-12-23 | 1995-05-30 | Universitge Laval | Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence |
US5436150A (en) | 1992-04-03 | 1995-07-25 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease |
US5356802A (en) | 1992-04-03 | 1994-10-18 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease |
US5487994A (en) | 1992-04-03 | 1996-01-30 | The Johns Hopkins University | Insertion and deletion mutants of FokI restriction endonuclease |
US5792632A (en) | 1992-05-05 | 1998-08-11 | Institut Pasteur | Nucleotide sequence encoding the enzyme I-SceI and the uses thereof |
US5587308A (en) | 1992-06-02 | 1996-12-24 | The United States Of America As Represented By The Department Of Health & Human Services | Modified adeno-associated virus vector capable of expression from a novel promoter |
EP0604662B1 (en) | 1992-07-07 | 2008-06-18 | Japan Tobacco Inc. | Method of transforming monocotyledon |
DE69534629D1 (de) | 1994-01-18 | 2005-12-29 | Scripps Research Inst | Derivate von zinkfingerproteinen und methoden |
US6242568B1 (en) | 1994-01-18 | 2001-06-05 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
US6140466A (en) | 1994-01-18 | 2000-10-31 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
ATE410518T1 (de) | 1994-03-23 | 2008-10-15 | Univ Ohio | Kompaktnukleinsäure und ihre verabreichung in zellen |
US5585245A (en) | 1994-04-22 | 1996-12-17 | California Institute Of Technology | Ubiquitin-based split protein sensor |
GB9824544D0 (en) | 1998-11-09 | 1999-01-06 | Medical Res Council | Screening system |
USRE39229E1 (en) * | 1994-08-20 | 2006-08-08 | Gendaq Limited | Binding proteins for recognition of DNA |
US5789538A (en) | 1995-02-03 | 1998-08-04 | Massachusetts Institute Of Technology | Zinc finger proteins with high affinity new DNA binding specificities |
US5928638A (en) | 1996-06-17 | 1999-07-27 | Systemix, Inc. | Methods for gene transfer |
US5925523A (en) | 1996-08-23 | 1999-07-20 | President & Fellows Of Harvard College | Intraction trap assay, reagents and uses thereof |
GB9703369D0 (en) | 1997-02-18 | 1997-04-09 | Lindqvist Bjorn H | Process |
GB2338237B (en) | 1997-02-18 | 2001-02-28 | Actinova Ltd | In vitro peptide or protein expression library |
US6342345B1 (en) | 1997-04-02 | 2002-01-29 | The Board Of Trustees Of The Leland Stanford Junior University | Detection of molecular interactions by reporter subunit complementation |
GB9710807D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
GB9710809D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
US5994136A (en) | 1997-12-12 | 1999-11-30 | Cell Genesys, Inc. | Method and means for producing high titer, safe, recombinant lentivirus vectors |
US6506559B1 (en) | 1997-12-23 | 2003-01-14 | Carnegie Institute Of Washington | Genetic inhibition by double-stranded RNA |
AR014491A1 (es) | 1998-01-29 | 2001-02-28 | Dow Agrosciences Llc | Metodo para obtener plantas transgenicas fertiles de gossypium hirsutum. |
US6410248B1 (en) | 1998-01-30 | 2002-06-25 | Massachusetts Institute Of Technology | General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites |
WO1999045132A1 (en) * | 1998-03-02 | 1999-09-10 | Massachusetts Institute Of Technology | Poly zinc finger proteins with improved linkers |
US6140081A (en) * | 1998-10-16 | 2000-10-31 | The Scripps Research Institute | Zinc finger binding domains for GNN |
US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
US7013219B2 (en) * | 1999-01-12 | 2006-03-14 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
US6534261B1 (en) * | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
US6503717B2 (en) * | 1999-12-06 | 2003-01-07 | Sangamo Biosciences, Inc. | Methods of using randomized libraries of zinc finger proteins for the identification of gene function |
US6794136B1 (en) | 2000-11-20 | 2004-09-21 | Sangamo Biosciences, Inc. | Iterative optimization in the design of binding proteins |
US7257541B1 (en) | 1999-10-08 | 2007-08-14 | I2 Technologies Us, Inc. | System and method for performing a business process in a multi-enterprise, collaborating network |
AU2001226935B2 (en) | 2000-01-24 | 2006-06-22 | Gendaq Limited | Nucleic acid binding polypeptides characterized by flexible linkers connected nucleic acid binding modules |
WO2001059450A2 (en) | 2000-02-08 | 2001-08-16 | Sangamo Biosciences, Inc. | Cells expressing zinc finger protein for drug discovery |
US20020061512A1 (en) | 2000-02-18 | 2002-05-23 | Kim Jin-Soo | Zinc finger domains and methods of identifying same |
WO2001088197A2 (en) | 2000-05-16 | 2001-11-22 | Massachusetts Institute Of Technology | Methods and compositions for interaction trap assays |
JP2002060786A (ja) | 2000-08-23 | 2002-02-26 | Kao Corp | 硬質表面用殺菌防汚剤 |
US9234187B2 (en) | 2001-01-22 | 2016-01-12 | Sangamo Biosciences, Inc. | Modified zinc finger binding proteins |
GB0108491D0 (en) | 2001-04-04 | 2001-05-23 | Gendaq Ltd | Engineering zinc fingers |
JP2005500061A (ja) | 2001-08-20 | 2005-01-06 | ザ スクリップス リサーチ インスティテュート | Cnnについての亜鉛フィンガー結合ドメイン |
AU2003251286B2 (en) | 2002-01-23 | 2007-08-16 | The University Of Utah Research Foundation | Targeted chromosomal mutagenesis using zinc finger nucleases |
EP2368982A3 (en) | 2002-03-21 | 2011-10-12 | Sangamo BioSciences, Inc. | Methods and compositions for using zinc finger endonucleases to enhance homologous recombination |
WO2004037977A2 (en) * | 2002-09-05 | 2004-05-06 | California Institute Of Thechnology | Use of chimeric nucleases to stimulate gene targeting |
US7888121B2 (en) | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
US8409861B2 (en) | 2003-08-08 | 2013-04-02 | Sangamo Biosciences, Inc. | Targeted deletion of cellular DNA sequences |
CA2534296C (en) | 2003-08-08 | 2013-03-26 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
US20070134796A1 (en) | 2005-07-26 | 2007-06-14 | Sangamo Biosciences, Inc. | Targeted integration and expression of exogenous nucleic acid sequences |
US7972854B2 (en) * | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
FR2872170B1 (fr) | 2004-06-25 | 2006-11-10 | Centre Nat Rech Scient Cnrse | Lentivirus non interactif et non replicatif, preparation et utilisations |
US20080131962A1 (en) * | 2006-05-25 | 2008-06-05 | Sangamo Biosciences, Inc. | Engineered cleavage half-domains |
US20060063231A1 (en) | 2004-09-16 | 2006-03-23 | Sangamo Biosciences, Inc. | Compositions and methods for protein production |
US20090263900A1 (en) | 2008-04-14 | 2009-10-22 | Sangamo Biosciences, Inc. | Linear donor constructs for targeted integration |
US8021867B2 (en) | 2005-10-18 | 2011-09-20 | Duke University | Rationally-designed meganucleases with altered sequence specificity and DNA-binding affinity |
WO2007139982A2 (en) | 2006-05-25 | 2007-12-06 | Sangamo Biosciences, Inc. | Methods and compositions for gene inactivation |
CN101679977B (zh) | 2007-04-26 | 2013-05-01 | 桑格摩生物科学股份有限公司 | 向ppp1r12c基因座的靶向整合 |
WO2009042186A2 (en) * | 2007-09-27 | 2009-04-02 | Sangamo Biosciences, Inc. | Genomic editing in zebrafish using zinc finger nucleases |
US8722410B2 (en) | 2007-10-05 | 2014-05-13 | Dow Agrosciences, Llc. | Methods for transferring molecular substances into plant cells |
EP2205752B1 (en) * | 2007-10-25 | 2016-08-10 | Sangamo BioSciences, Inc. | Methods and compositions for targeted integration |
US8029993B2 (en) * | 2008-04-30 | 2011-10-04 | Population Genetics Technologies Ltd. | Asymmetric adapter library construction |
JP2011521643A (ja) | 2008-05-28 | 2011-07-28 | サンガモ バイオサイエンシーズ, インコーポレイテッド | Dna結合ドメインおよび切断ドメインを連結するための組成物 |
CA2741119C (en) * | 2008-10-29 | 2019-02-12 | Sangamo Biosciences, Inc. | Methods and compositions for inactivating glutamine synthetase gene expression |
EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
GB0905458D0 (en) * | 2009-03-30 | 2009-05-13 | Gene Bridges Gmbh | Method of altering nucleic acids |
US8586363B2 (en) * | 2009-12-10 | 2013-11-19 | Regents Of The University Of Minnesota | TAL effector-mediated DNA modification |
EP3156062A1 (en) * | 2010-05-17 | 2017-04-19 | Sangamo BioSciences, Inc. | Novel dna-binding proteins and uses thereof |
US9405700B2 (en) | 2010-11-04 | 2016-08-02 | Sonics, Inc. | Methods and apparatus for virtualization in an integrated circuit |
-
2011
- 2011-02-09 JP JP2012552874A patent/JP2013518602A/ja active Pending
- 2011-02-09 US US12/931,760 patent/US9255259B2/en active Active
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- 2011-02-09 EP EP11742576.9A patent/EP2534163B1/en active Active
- 2011-02-09 EP EP13166487.2A patent/EP2660318A1/en not_active Withdrawn
- 2011-02-09 CA CA2788850A patent/CA2788850C/en active Active
- 2011-02-09 WO PCT/US2011/000245 patent/WO2011100058A1/en active Application Filing
- 2011-07-08 US US13/135,558 patent/US9005973B2/en active Active
-
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- 2013-06-05 HK HK13106603.1A patent/HK1179628A1/zh not_active IP Right Cessation
-
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- 2016-01-07 US US14/990,300 patent/US9970028B2/en active Active
- 2016-02-12 AU AU2016200945A patent/AU2016200945A1/en not_active Abandoned
Patent Citations (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2010006237A2 (en) * | 2008-07-11 | 2010-01-14 | Alnylam Pharmaceuticals, Inc. | Phosphorothioate oligonucleotides non-nucleosidic phosphorothiotes as delivery agents for irna agents |
WO2010143917A2 (en) * | 2009-06-11 | 2010-12-16 | Toolgen Incorporation | Targeted genomic rearrangements using site-specific nucleases |
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